Today, on International Arginase 1 Deficiency (ARG1-D) Day, we join families, patients, clinicians, researchers, and advocates around the world to raise awareness of this ultra-rare disease and celebrate a year of extraordinary progress. For decades, families affected by ARG1-D faced a future focused primarily on managing symptoms, slowing progression, and preserving mobility and cognitive function [...]
In Honor of JOSH on RARE DISEASE DAY A Mother's Story Joshua Eaton, Pioneer in the ARG1-D Community, Age 27 South Shore, Massachusetts We share Josh's story so his life can inspire others – to support families facing Arginase 1 Deficiency, to raise awareness, and to help ensure no child loses precious time waiting for [...]
Yesterday, the U.S. Food and Drug Administration (FDA) approved Pegzilarginase (Loargys) — the first and only treatment for Arginase 1 Deficiency (ARG1-D). This is not just a milestone. This is hope realized. For families living with ARG1-D, this approval represents something we have waited for, worked toward, advocated for, and prayed over for years — [...]
Dear Arginase 1 Deficiency Community & Families, If you have not seen already, we are excited to share that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Loargys® (pegzilarginase-nbln), a prescription medicine used to treat high levels of arginine in the blood of adults and children 2 years and older with [...]
Dear Friends, Sponsors, and Hope on the Court Community, From the bottom of our hearts, thank you for making the Hope on the Court Pickleball Tournament such an extraordinary and meaningful day. Because of you, this event was far more than a tournament—it was a celebration of connection, generosity, and purpose.
The National Urea Cycle Disorders Foundation (NUCDF) is pleased to announce that it has awarded the 2025 Cynthia Le Mons Fellowship to Shradha Suyal, PhD, a postdoctoral fellow at the Pacific Northwest Research Institute (PNRI). Suyal (pictured above, left) works in the lab of Aimée Dudley, PhD (pictured above, right), who uses yeast genetics to study the severity [...]
Dr. Stephen Cederbaum, a longtime UCLA Health physician and renowned expert in genetic medicine, has been awarded the 2025 David L. Rimoin Lifetime Achievement Award by the ACMG Foundation for Genetic and Genomic Medicine. The award honors professionals who have dedicated their career to advancing science and improving lives.
Stockholm, November 5, 2024: Immedica announces today that the Biologics License Application (BLA) for pegzilarginase in the treatment of arginase 1 deficiency (ARG1-D) has been successfully validated and accepted for priority review by the U.S. Food and Drug Administration (FDA). Anders Edvell, CEO of Immedica commented: “We are pleased that the FDA has accepted the [...]
Loargys is approved in the EU and Great Britain for the treatment of ARG1-D in adults, adolescents and children aged 2 years and older. The initiation of a clinical study in children below 2 years of age is of high importance in this progressive disease exposing patients to elevated toxic levels of arginine from birth.